The US Food and Drug Administration (FDA) has directed considerable effort towards modernizing its regulatory processes over the past decade to address the challenges in the drug development sector. Through partnerships and input from stakeholders, multiple initiatives are under way, many projects have been launched, several have resulted in tangible results, and many are ongoing and under discussion. We are learning that collaborative efforts can better inform and leverage existing knowledge, that the challenges of data sharing and intellectual property can be overcome, and that there is wide interest in partnering to address key public health regulatory science issues. It is crucial that we continue to build on these initial efforts to facilitate drug development.
Easy access to large quantities of accurate health data is required to understand medical and scientific information in real-time; evaluate public health measures before, during, and after times of crisis; and prevent medical errors. Introducing a system in the USA that allows for efficient access to such health data and ensures auditability of data facts, while avoiding data silos, will require fundamental changes in current practices. Here, we recommend the implementation of standardized data collection and transmission systems, universal identifiers for individual patients and end users, a reference standard infrastructure to support calibration and integration of laboratory results from equivalent tests, and modernized working practices. Requiring comprehensive and binding standards, rather than incentivizing voluntary and often piecemeal efforts for data exchange, will allow us to achieve the analytical information environment that patients need.
Since 2008, the Critical Path Initiative has supported FDA's program of intramural research projects in regulatory science, with the goal of improving translation of advances in emerging sciences to the development of safe and effective medical products. Since 2011, the research of FDA's Center for Drug Evaluation and Research (CDER), including the work supported by the Critical Path Initiative, has been guided by the regulatory science needs identified in the CDER science and research needs report. In this review, the authors highlight a few of CDER'S Critical Path Initiative research projects, each addressing a different regulatory science need, to illustrate the diversity of regulatory science at CDER. They also describe elements common to these research projects, including broad collaboration with external partners, an increasing dependence on large data sets and computational models, and requirements for resources or perspectives specific to FDA.
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